Rocket Pharma Says FDA Grants Fast Track And Orphan Drug Designations For RP-A601 Gene Therapy

RTTNews | 761 days ago
Stocktwits
Rocket Pharma Says FDA Grants Fast Track And Orphan Drug Designations For RP-A601 Gene Therapy

(RTTNews) - Biotechnology company Rocket Pharmaceuticals, Inc. (RCKT) announced Thursday that the U.S. Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to RP-A601, the Company's gene therapy candidate for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).

RP-A601 is Rocket's adeno-associated virus (AAV.rh74)-based gene therapy for PKP2-ACM, a devastating, inherited heart disease associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.

Rocket recently announced FDA clearance of the Investigational New Drug (IND) application for RP-A601 based on robust preclinical proof of concept studies that demonstrated decreased arrhythmias and increased survival.

The Company is initiating a multi-center Phase 1, dose escalation trial that will evaluate the safety and preliminary efficacy of RP-A601 in at least six adult PKP2-ACM patients with ICDs and who have overall high risk for life-threatening arrhythmias.

For More Such Health News, visit rttnews.com

Tags: RCKT
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UK Stocks Turning In Mixed Performance In Cautious Trade

UK Stocks Turning In Mixed Performance In Cautious Trade

U.K. stocks are turning in a mixed performance Tuesday morning as investors are treading cautiously, keeping track of U.S. President Donald Trump's tariff plans and the potential risks to global economic growth.
RTTNews | 2h 58min ago
CAC 40 Slightly Lower In Lackluster Trade

CAC 40 Slightly Lower In Lackluster Trade

French stocks are slightly lower on Tuesday with investors making cautious moves while closely following the developments on the trade front.
RTTNews | 3h 45min ago
Australia Central Bank Unexpectedly Maintains Status Quo

Australia Central Bank Unexpectedly Maintains Status Quo

The Reserve Bank of Australia adopted a wait-and-see approach on Tuesday, defying expectations for a quarter-point rate cut but confirmed that an easing cycle is coming. The policy board, governed by Michele Bullock, decided to hold the cash rate target at 3.85 percent. A majority of six members voted to hold the rate, while three members sought a change from 3.85 percent.
RTTNews | 3h 56min ago
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